ClinicalTrials.gg
TerminatedNCT03738800Updated Aug 14, 2023Results posted

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

A Phase 2 interventional study of CD5789 Cream 200 µg/g and CD5789 Cream 100 µg/g in Lamellar Ichthyosis, sponsored by Mayne Pharma International Pty Ltd. Terminated at 36 sites in 9 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2023-08-14.

Sponsored by Mayne Pharma International Pty Ltd · Phase 2, Interventional, and Treatment

Why this study was terminated
Futility
Phase
Phase 2
Study type
Interventional
Enrollment
65
Allocation
Randomized
Ages
12 Years and older
Sex
All
01

Study summary

This is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with autosomal recessive ichthyosis with lamellar scale.

Read the detailed description

This is a 2-cohort, multicenter study in subjects with moderate to severe LI. Adults (Cohort A) and adults and adolescents (Cohort B) will be randomized in a double-blind fashion to 1 of 2 doses of active or vehicle and treated twice weekly for 90 days. Subjects who complete the randomized, double-blind portion of the study will be eligible to enter a 90 day, open-label extension study.

Approximately 15 adults (≥18 years old) will be randomized into the first cohort of subjects (Cohort A) in a 1:1:1 ratio and treated twice weekly for up to 90 days. If no safety issues are identified, both adults and adolescents (ages 12-17 years, inclusive) will be allowed to enroll in Cohort B. Subjects in Cohort B will be randomized 1:1:1 and treated twice weekly for up to 90 days in the same manner as subjects in Cohort A.

All subjects who complete 90 days of double-blind study treatment will be eligible to enroll in a 90 open-label extension. Subjects in the open-label extension will receive active twice weekly for up to 90 days.

02

Conditions studied

  • Lamellar Ichthyosis

Browse trials for

03

In context

Ichthyosis, Lamellar

7 studies on the registry are indexed under Ichthyosis, Lamellar; 1 is open to participants now.

Browse Ichthyosis, Lamellar studies →

Lead sponsor

Mayne Pharma International Pty Ltd is the lead sponsor of 8 studies on the registry; none are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

  1. For Cohort A: subject is ≥18 years old; for Cohort B: subject is ≥12 years old.
  2. Subject has known diagnosis of LI.
  3. Subject has moderate to severe (IGA 3-4) LI on the IGA of LI severity.
  4. Subject has signed an ICF at Screening before any procedures. Subjects \<18 years of age (or Age of Majority) must sign an assent form in conjunction with an ICF signed by the parent/legal representative.
  5. Subject who is participating in optional photography has signed a photography ICF.
  6. Subject who is participating in the optional PK substudy has signed a PK ICF. Minors, in the event of their reaching majority during the study, should be capable of giving consent to take part in the PK substudy.
  7. Subject is not of , who is (absence of menstrual bleeding for 1 year before Baseline, without any other medical reason), or has documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy. For individuals with permanent infertility due to an alternate medical cause other than the above, (e.g., Mullerian agenesis, androgen insensitivity), investigator discretion should be applied to determining study entry.

    OR

    • Subject is a woman of childbearing potential (WOCBP), i.e., a female ≥12 years of age (regardless of whether they have experienced/reported menarche), or a male subject with sexual partners capable of reproduction who agrees to use 2 effective forms of during the study and for at least 1 month after the last study drug application. The 2 authorized forms of contraception are condom used with 1 of the following methods of contraception:
    • bilateral tubal ligation
    • combined oral contraceptives (estrogens and progesterone), vaginal ring, or implanted or injectable hormonal contraceptives with a stable dose for at least 1 month before Baseline; hormonal contraceptives must inhibit ovulation
    • intrauterine device (IUD) inserted at least 1 month before Baseline OR Agrees to abstain from heterosexual intercourse during study participation and for 1 month after the last application of study drug and to use a highly effective contraceptive as backup if he or she becomes sexually active during the study. Abstinence is only acceptable if this is the subject's usual lifestyle. Periodic abstinence (calendar, symptothermal, postovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception.

    AND Male subjects may not donate sperm during the study and for at least 1 month after the last study drug application.

    Note: Female subjects who are premenstrual at screening should nonetheless follow the pregnancy testing schedule for WOCBP even if they abstain from sexual intercourse while in the study and for at least 1 month after the last study drug application.

  8. Women of childbearing potential must be nonlactating and have negative pregnancy test results at Screening (serum) and on Day 1 before study drug administration (urine).
  9. Subject is reliable and capable of adhering to the protocol and visit schedule, in the investigator's judgment, and has signed /assent, as applicable.
  10. Subject is taking no more than 3500 IU/day Vitamin A (e.g., as in a multivitamin).

Exclusion criteria:

  1. Subject has any variant of ichthyosis other than LI or another disorder of keratinization, including syndromic ichthyoses.
  2. Subject has current moderate or severe stinging/burning at Screening.
  3. Subject has an ongoing cutaneous infection or any other significant concomitant skin disease (other than the LI) which, in the investigator's opinion, may interfere with the study assessments.
  4. Subject with fasting triglycerides >200 mg/dL or >2.25 mmol/L and/or total cholesterol >250 mg/dL or >6.5 mmol/L. Subjects whose triglycerides and/or total cholesterol are within normal limits with a stable dose of lipid-lowering agents for at least 6 months may be included.
  5. Subject was previously treated with trifarotene/CD5789 in an acne or ichthyosis study.
  6. Subject has any other significant concomitant disease, or poorly controlled medical condition other than LI that in the investigator's opinion may put him or her at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
  7. Subject has a medical condition that potentially alters bone metabolism (e.g., osteoporosis, thyroid dysfunction, Cushing syndrome, Crohn's disease, or ulcerative colitis). Subjects with hypothyroidism who are on a stable dose of thyroid hormone replacement therapy and whose thyroid-stimulating hormone (TSH) is normal may be included
  8. Subject is being treated for major depression disorder and/or has a history of major depression or suicide attempt requiring hospitalization, medications, and close psychiatric surveillance to prevent suicide attempts.
  9. Subject with positive serology for surface antigen, , or are known to be positive or to have AIDS at Screening.
  10. Subject with any of the following laboratory values at Screening:

    1. or alanine aminotransferase >1.5 × defined by the laboratory
    2. Total >1.25 × ULN at Screening. Subjects with known Gilbert's syndrome may be included with total bilirubin >1.25 × ULN
    3. \<12.5 g/dL for men and \<11.5 g/dL for women
    4. \<150 × 109/L or >400 × 109/L.
  11. Subject has any clinically other significant abnormal laboratory value (hematology, chemistry, or urinalysis) at Screening that, in the investigator's opinion, may put the subject at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
  12. Subject has had recent systemic (e.g., within 5 years) with exception of nonmelanoma skin cancer or cervical intraepithelial neoplasia of Grade 1 who are >6 months post-treatment.
  13. Subject has a history of long syndrome or has (ECG) abnormalities, including clinically significant conduction disorders or significant arrhythmias, or QTcF interval >450 ms.
  14. Subject has a known allergy or sensitivity to any of the components of the investigational products.
  15. Subject has been exposed to excessive UV radiations on the treated zones within 1 month before Baseline visit or is planning intensive UV exposure during the study (e.g., occupational exposure to the sun, sunbathing, phototherapy, etc.).
  16. Subject is inherently sensitive to sunlight.
  17. Subject is unable or unwilling to stop use of topical or systemic retinoids.
  18. Subject is presumed to be abusing drug or alcohol at Screening or Baseline Visits based on medical history or current clinical symptoms.
  19. Subject is participating in another interventional clinical trial.
  20. Subject is institutionalized.
  21. Subject is in any way related to the sponsor, investigator, or site personnel.
Terms in these criteria, in plain language(17)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
65 participants (actual)

Study arms

  • Experimental
    CD5789 Cream 200 µg/g

    CD5789 200 µg/g, topical, 50g

    Drug: CD5789 Cream 200 µg/g

  • Experimental
    CD5789 Cream 100 µg/g

    CD5789 100 µg/g, topical, 50g

    Drug: CD5789 Cream 100 µg/g

  • Placebo comparator
    CD5789 Cream Vehicle

    CD5789 Cream Vehicle, topical, 50g

    Drug: CD5789 Cream Vehicle

Interventions

  • DrugCD5789 Cream 200 µg/g

    Drug page: Trifarotene

    A fixed dose (determined at Visit 1) of 200 µg/g applied topically twice weekly to up to 90% BSA

  • DrugCD5789 Cream 100 µg/g

    Drug page: Trifarotene

    A fixed dose (determined at Visit 1) of 100 µg/g applied topically twice weekly to up to 90% BSA

  • DrugCD5789 Cream Vehicle

    A fixed dose (determined at Visit 1) applied topically twice weekly, up to 36 g per dose up to 90% BSA

06

What researchers measure

Primary outcomes

  1. The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.

    The percentage of subjects in each treatment group who experienced successful resolution of LI where "success" is defined as clear/almost clear on treated areas and at least a 2-grade change from Baseline at Day 90/end-of-treatment (EOT) in the Double-blind Period on the 5-point IGA full body scale.

    Time frame: 90 Days

Secondary outcomes

  1. Total 16-point Visual Index for Ichthyosis Severity (VIIS)

    5-point Visual Index for Ichthyosis Severity (VIIS) for scaling (overall 16 points) for scaling, i.e. 0-4 points for 4 body areas: chest/abdomen, back, arms and legs) where minimum is 0 and maximum is 16 (e.g. 4 points for each of the four body parts). 0 (Clear) No scaling 1. (Almost Clear) Very fine, non-coalescent scales 2. (Mild) Small and thin, non-coalescent scales 3. (Moderate) Large and rather thick scales starting to coalesce 4. (Severe) Very large, adherent, coalescent and very thick scales

    Time frame: 90 Days

  2. The Difference in Mean Scores Using Individual Score for Roughness

    The amount of roughness of the skin will be measured on a 5-point scale. 0 (Clear) Smooth skin 1. (Almost Clear) Hardly palpably roughness 2. (Mild) Mild roughness (fine sand paper-like) 3. (Moderate) Moderate, coarse roughness (coarse sand paper-like) 4. (Severe) Very coarse skin (broken cornflakes-like)

    Time frame: 90 Days

  3. The Difference in Mean Scores Using Palm Sole Assessment

    Thickening of the skin on the palms and soles will be measured on a 5-point scale: 0 (Clear) No thickening, no roughness, no fissure 1. (Almost Clear) Only slight thickening, minimal to no roughness, no fissures 2. (Mild) Some thickening, mild roughness on palpation, few fissures may be present 3. (Moderate) Substantial and diffuse thickening, coarse roughness on palpation may be present, fissures may be present 4. (Severe) Very thickened and rough skin, numerous fissures

    Time frame: 90 Days

  4. The Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups

    Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups

    Time frame: 90 Days

  5. Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)

    The DLQI, or the Dermatology Quality of Life Index, is a dermatology-specific Quality of Life instrument. It is a simple 10-question validated questionnaire with 6 domains (symptoms and feelings, daily activities, leisure, work and school, personal relationships, and treatment); higher scores indicate poorer quality of life. Responses collected are on a scale of 0-3 depending on the question relevance to the subject. Response (Score) Very much (scored 3) A lot (scored 2) A little (scored 1) Not at all (scored 0) Not relevant (scored 0) A minimum score of 0 and maximum score of 30 is obtained by summing the score of each question. The higher the score, the more quality of life is impaired. 0-1 = no effect at all on patient's life 2-5 = small effect on patient's life 6-10 = moderate effect on patient's life 11-20 = very large effect on patient's life 21-30 = extremely large effect on patient's life

    Time frame: 90 Days

  6. The Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups

    Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups

    Time frame: 90 Days

Other outcomes

  1. The Difference in Mean Ectropion Scores Between the Active and Vehicle Groups

    The Ectropion Severity Score (ESS), is a proven system to be reliable and sensitive to the presence of ectropion and has a maximum score of 8 points (0-8). A higher score indicates a worse ectropion. The score takes the severity of ectropion in terms of lateral and medial apposition, scleral show, conjunctival show, and roundness of the eye into account and gives an indication of the functional aspects involved in ectropion by scoring redness, excess tear film, and the position of the lacrimal punctum A point scale of 0=Nonaffected, 0.5=Emerging, 1= Affected is assigned to 8 observations. * Lateral apposition * Medial apposition * Sceral show * Conjunctival show * Excess team film * Redness of the eye * Round canthus * Punctum lacrimale

    Time frame: 180 Days

  2. Quality of Life Measurement Per EQ-5D-5L

    The EQ-5D is a standardized instrument developed by the EuroQol Group as a measure of health-related quality of life used in a wide range of health conditions and treatments. The EQ-5D consists of a descriptive system and the EQ visual analog scale (VAS). Descriptive system of health-related quality of life states consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) each of which can take 1 of 5 responses. 1. no problem 2. slight problems 3. moderate problems 4. severe problems 5. extreme problems It should be noted that the numerals 1-5 have no arithmetic properties and should not be used as a cardinal score. The EQ VAS records the respondent's self-rated health on a 20 cm vertical, visual analogue scale with endpoints labelled 100-'the best health you can imagine' and 0-'the worst health you can imagine'. This information can be used as a quantitative measure of health as judged by the individual respondents.

    Time frame: 180 Days

  3. Incidents of Adverse Events

    The number of subjects with AEs will be collected for each treatment group

    Time frame: 180 Days

  4. Measurement of Local Tolerability

    Local tolerability will be assessed on a 0-3 scale (none, mild, moderate, severe).

    Time frame: 180 Days

  5. Clinical Laboratory Evaluations

    The number of subjects with clinical laboratory values categorized as below (vital signs, 12 lead electrocardiogram, Physical Exam), within, or above normal ranges will be evaluated (changes from baseline for each clinical laboratory parameter by treatment group and by study visit).

    Time frame: 180 Days

  6. Measurement of Vital Signs

    Blood pressure (systolic blood pressure \[SBP\], diastolic blood pressure \[DBP\] and pulse will be measured. Measurement of actual values and changes from baseline will be calculated. Vital signs The number of subjects with vital signs values categorized as below, within, or above normal ranges (change from baseline for each parameter by period, by treatment group and by study visit).

    Time frame: 180 Days

  7. Measurement of 12-lead ECG Readings

    The number of subjects with normal and abnormal ECG findings will be measured for each treatment group at each time point for QT and the QT interval corrected for heart rate (QTc) calculated using Fridericia's QT correction methods.

    Time frame: 180 Days

  8. Physical Examination Findings

    The number of subjects with normal and abnormal findings in the complete physical examination for each treatment group. This is a limited physical examination to include HEENT, cardiorespiratory, abdomen, and range of motion.

    Time frame: 180 Days

  9. Measurement of Area Under the Plasma Concentration Versus Time Curve (AUC)

    Measurement of the extent of absorption using estimates of the area-under-the-curve (AUC).

    Time frame: 180 Days

  10. Measurement of Peak Plasma Concentration (Cmax)

    Measurement of therate-of-absorption using the maximum concentration (Cmax) and the time of Cmax (Tmax).

    Time frame: 180 Days

07

Results

Posted Aug 14, 2023

Participant flow

Double-blind Treatment Period
Participant flow — Double-blind Treatment Period
MilestoneCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream Vehicle
Started232121
Completed141817
Not completed934
Withdrew: Withdrawal by subject200
Withdrew: Study discontinued by sponsor533
Withdrew: Lack of efficacy201
Open Label Extension
Participant flow — Open Label Extension
MilestoneCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream Vehicle
Started4900
Completed3900
Not completed1000

Outcome measures

PrimaryThe Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.

The percentage of subjects in each treatment group who experienced successful resolution of LI where "success" is defined as clear/almost clear on treated areas and at least a 2-grade change from Baseline at Day 90/end-of-treatment (EOT) in the Double-blind Period on the 5-point IGA full body scale.

Time frame:
90 Days
Reported as:
Count of participants · Participants
The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.
ParticipantsCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream Vehicle
The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.020
SecondaryTotal 16-point Visual Index for Ichthyosis Severity (VIIS)

5-point Visual Index for Ichthyosis Severity (VIIS) for scaling (overall 16 points) for scaling, i.e. 0-4 points for 4 body areas: chest/abdomen, back, arms and legs) where minimum is 0 and maximum is 16 (e.g. 4 points for each of the four body parts). 0 (Clear) No scaling 1. (Almost Clear) Very fine, non-coalescent scales 2. (Mild) Small and thin, non-coalescent scales 3. (Moderate) Large and rather thick scales starting to coalesce 4. (Severe) Very large, adherent, coalescent and very thick scales

Time frame:
90 Days
Reported as:
Mean · units on a scale
Total 16-point Visual Index for Ichthyosis Severity (VIIS)
units on a scaleCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
Total 16-point Visual Index for Ichthyosis Severity (VIIS)10.9 ± 4.2410.9 ± 3.5110.9 ± 3.8611.4 ± 2.94
SecondaryThe Difference in Mean Scores Using Individual Score for Roughness

The amount of roughness of the skin will be measured on a 5-point scale. 0 (Clear) Smooth skin 1. (Almost Clear) Hardly palpably roughness 2. (Mild) Mild roughness (fine sand paper-like) 3. (Moderate) Moderate, coarse roughness (coarse sand paper-like) 4. (Severe) Very coarse skin (broken cornflakes-like)

Time frame:
90 Days
Reported as:
Mean · score on a scale
The Difference in Mean Scores Using Individual Score for Roughness
score on a scaleCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
The Difference in Mean Scores Using Individual Score for Roughness2.7 ± 1.102.7 ± 0.992.7 ± 1.023.1 ± 0.78
SecondaryThe Difference in Mean Scores Using Palm Sole Assessment

Thickening of the skin on the palms and soles will be measured on a 5-point scale: 0 (Clear) No thickening, no roughness, no fissure 1. (Almost Clear) Only slight thickening, minimal to no roughness, no fissures 2. (Mild) Some thickening, mild roughness on palpation, few fissures may be present 3. (Moderate) Substantial and diffuse thickening, coarse roughness on palpation may be present, fissures may be present 4. (Severe) Very thickened and rough skin, numerous fissures

Time frame:
90 Days
Reported as:
Mean · score on a scale
The Difference in Mean Scores Using Palm Sole Assessment
score on a scaleCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
The Difference in Mean Scores Using Palm Sole Assessment2.1 ± 1.061.6 ± 1.271.9 ± 1.182.6 ± 1.06
SecondaryThe Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups

Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups

Time frame:
90 Days
Reported as:
Count of participants · Participants
The Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups
ParticipantsCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
Number of participants with Fissures Present at Day 905497
Number of participants with no fissures present at day 9010122210
SecondaryQuality of Life Measurement Per Dermatology Life Quality Index (DLQI)

The DLQI, or the Dermatology Quality of Life Index, is a dermatology-specific Quality of Life instrument. It is a simple 10-question validated questionnaire with 6 domains (symptoms and feelings, daily activities, leisure, work and school, personal relationships, and treatment); higher scores indicate poorer quality of life. Responses collected are on a scale of 0-3 depending on the question relevance to the subject. Response (Score) Very much (scored 3) A lot (scored 2) A little (scored 1) Not at all (scored 0) Not relevant (scored 0) A minimum score of 0 and maximum score of 30 is obtained by summing the score of each question. The higher the score, the more quality of life is impaired. 0-1 = no effect at all on patient's life 2-5 = small effect on patient's life 6-10 = moderate effect on patient's life 11-20 = very large effect on patient's life 21-30 = extremely large effect on patient's life

Time frame:
90 Days
Reported as:
Mean · score on a scale
Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)
score on a scaleCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)6.1 ± 5.225.9 ± 5.216.0 ± 5.116.2 ± 5.78
SecondaryThe Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups

Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups

Time frame:
90 Days
Reported as:
Count of participants · Participants
The Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups
ParticipantsCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gOverall Trifarotene Cream (200 µg/g and 100 µg/g)Vehicle Cream
Numer of participants with fissures on soles present at day 905497
Numer of participants with no fissures on soles present at day 9010122210
Other pre-specifiedThe Difference in Mean Ectropion Scores Between the Active and Vehicle Groups

The Ectropion Severity Score (ESS), is a proven system to be reliable and sensitive to the presence of ectropion and has a maximum score of 8 points (0-8). A higher score indicates a worse ectropion. The score takes the severity of ectropion in terms of lateral and medial apposition, scleral show, conjunctival show, and roundness of the eye into account and gives an indication of the functional aspects involved in ectropion by scoring redness, excess tear film, and the position of the lacrimal punctum A point scale of 0=Nonaffected, 0.5=Emerging, 1= Affected is assigned to 8 observations. * Lateral apposition * Medial apposition * Sceral show * Conjunctival show * Excess team film * Redness of the eye * Round canthus * Punctum lacrimale

Time frame:
180 Days
Reported as:
Mean · score on a scale
The Difference in Mean Ectropion Scores Between the Active and Vehicle Groups
score on a scaleCD5789 Cream 200 µg/g
The Difference in Mean Ectropion Scores Between the Active and Vehicle Groups2.82 ± 3.077
Other pre-specifiedQuality of Life Measurement Per EQ-5D-5L

The EQ-5D is a standardized instrument developed by the EuroQol Group as a measure of health-related quality of life used in a wide range of health conditions and treatments. The EQ-5D consists of a descriptive system and the EQ visual analog scale (VAS). Descriptive system of health-related quality of life states consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) each of which can take 1 of 5 responses. 1. no problem 2. slight problems 3. moderate problems 4. severe problems 5. extreme problems It should be noted that the numerals 1-5 have no arithmetic properties and should not be used as a cardinal score. The EQ VAS records the respondent's self-rated health on a 20 cm vertical, visual analogue scale with endpoints labelled 100-'the best health you can imagine' and 0-'the worst health you can imagine'. This information can be used as a quantitative measure of health as judged by the individual respondents.

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedIncidents of Adverse Events

The number of subjects with AEs will be collected for each treatment group

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedMeasurement of Local Tolerability

Local tolerability will be assessed on a 0-3 scale (none, mild, moderate, severe).

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedClinical Laboratory Evaluations

The number of subjects with clinical laboratory values categorized as below (vital signs, 12 lead electrocardiogram, Physical Exam), within, or above normal ranges will be evaluated (changes from baseline for each clinical laboratory parameter by treatment group and by study visit).

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedMeasurement of Vital Signs

Blood pressure (systolic blood pressure \[SBP\], diastolic blood pressure \[DBP\] and pulse will be measured. Measurement of actual values and changes from baseline will be calculated. Vital signs The number of subjects with vital signs values categorized as below, within, or above normal ranges (change from baseline for each parameter by period, by treatment group and by study visit).

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedMeasurement of 12-lead ECG Readings

The number of subjects with normal and abnormal ECG findings will be measured for each treatment group at each time point for QT and the QT interval corrected for heart rate (QTc) calculated using Fridericia's QT correction methods.

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedPhysical Examination Findings

The number of subjects with normal and abnormal findings in the complete physical examination for each treatment group. This is a limited physical examination to include HEENT, cardiorespiratory, abdomen, and range of motion.

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedMeasurement of Area Under the Plasma Concentration Versus Time Curve (AUC)

Measurement of the extent of absorption using estimates of the area-under-the-curve (AUC).

Time frame:
180 Days

Results for this outcome have not been posted.

Other pre-specifiedMeasurement of Peak Plasma Concentration (Cmax)

Measurement of therate-of-absorption using the maximum concentration (Cmax) and the time of Cmax (Tmax).

Time frame:
180 Days

Results for this outcome have not been posted.

Adverse events

Collected over The maximum study duration for each subject was approximately 291 days.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
CD5789 Cream 200 µg/g0/23 (0%)0/23 (0%)10/23 (43.5%)
CD5789 Cream 100 µg/g0/21 (0%)0/21 (0%)7/21 (33.3%)
CD5789 Cream Vehicle0/21 (0%)0/21 (0%)11/21 (52.4%)
Most frequent other events
Showing 10 of 36
Most frequent other events
EventCD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream Vehicle
DysmenorrhoeaReproductive system and breast disorders1/231/213/21
HeadacheNervous system disorders3/231/212/21
Back painMusculoskeletal and connective tissue disorders2/231/212/21
NasopharyngitisInfections and infestations0/230/212/21
Arthropod biteInjury, poisoning and procedural complications2/232/210/21
PruritusSkin and subcutaneous tissue disorders2/230/210/21
EczemaSkin and subcutaneous tissue disorders0/231/211/21
DermatitisSkin and subcutaneous tissue disorders0/231/210/21
Dermatitis contactSkin and subcutaneous tissue disorders0/230/211/21
Pain in extremityMusculoskeletal and connective tissue disorders0/231/211/21

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
<=18 years84113
Between 18 and 65 years15141948
>=65 years0314
Age, Continuous
Age, Continuous(years)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
Mean32.1 ± 17.0835.1 ± 20.7031.5 ± 13.3432.9 ± 17.09
Sex: Female, Male
Sex: Female, Male(Participants)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
Female11161441
Male125724
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
Hispanic or Latino0202
Not Hispanic or Latino23172060
Unknown or Not Reported0213
Race (NIH/OMB)
Race (NIH/OMB)(Participants)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
American Indian or Alaska Native0000
Asian0134
Native Hawaiian or Other Pacific Islander0000
Black or African American3148
White18151346
More than one race1102
Unknown or Not Reported1315
5-point Investigator's Global Assessment [IGA]
5-point Investigator's Global Assessment [IGA](units on a scale)CD5789 Cream 200 µg/gCD5789 Cream 100 µg/gCD5789 Cream VehicleTotal
Mean3.5 ± 0.513.5 ± 0.513.3 ± 0.483.4 ± 0.50
08

Study locations

36 sites
  • TCR Medical Corporation
    San Diego, California 92123, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Yale University
    New Haven, Connecticut 06519, United States
  • NorthShore University HealthSystem
    Skokie, Illinois 60077, United States
  • Dawes Fretzin Clinical Research Group, LLC
    Indianapolis, Indiana 46250, United States
  • DermAssociates, PC
    Rockville, Maryland 20850, United States
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Medical University of South Carolina
    Charleston, South Carolina 29425, United States
  • Texas Dermatology and Laser Specialists
    San Antonio, Texas 78218, United States
  • Eastern Health Monash University
    Box Hill, Australia
  • Veracity Clinical Research
    Brisbane, Australia
  • Royal Children's Hospital
    Parkville, Australia
  • Premier Specialists Ptd Ltd
    Sydney, Australia
  • The Hospital for Sick Children
    Toronto, Canada
  • Dermatologie pédiatrique
    Paris, France
  • CHU Charles Nicolle
    Rouen, France
  • CHU de Toulouse- Hospital Larrey
    Toulouse, France
  • Charite - Universitaetsmedizin Berlin
    Berlin, Germany
  • Universitätsklinkum Frankfurt
    Frankfurt, Germany
  • Kath. Kinderkrankenhaus Wilhelmstift
    Hamburg, Germany
  • Ludwig-Maximilians University
    Munich, Germany
  • Universitatsmedizin Rostock
    Rostock, Germany
  • Tel Aviv Sourasky Mc
    Tel Aviv, Israel
  • Hospital Clinic de Barcelona
    Barcelona, Spain
  • Hospital Nino Jesus
    Madrid, 28009, Spain
  • Clinica Universidad de Navarra (Madrid)
    Madrid, Spain
  • Hospital Niño Jesús
    Madrid, Spain
  • Clinica Universidad de Navarra
    Pamplona, Spain
  • Medical Center of Private Enterprise "Dzerkalo"
    Dnipro, 49000, Ukraine
  • Dnipropetrovsk State Hospital of Dermatovenerology
    Dnipro, 49074, Ukraine
  • Medical Center "Family Medicine Clinic"
    Dnipro, 49600, Ukraine
  • Ternopil Regional Clinical Dermatovenereological Dispensary
    Ternopil', 46006, Ukraine
  • TDC PE "Asclepius"
    Uzhhorod, 88000, Ukraine
  • Community Institution "Zaporizhzhya Regional Dermatovenereology Clinical Hospital"
    Zaporizhzhya, 69063, Ukraine
  • Royal London Hospital Barts Health Nhs Trust
    London, United Kingdom
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 14, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03738800
Lead sponsor
Mayne Pharma International Pty Ltd
Responsible party
Sponsor
First posted
Nov 13, 2018
Start date
May 1, 2019
Primary completion
Sep 3, 2021
Completion
Sep 3, 2021
Results posted
Aug 14, 2023
Last update
Aug 14, 2023

Study contacts

Keith A. Choate, MD
principal investigator · Yale University

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Feb 2023. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: Trifarotene

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